Mar. 11The Food and Drug Administration announced Tuesday that some patients with a degenerative nerve disease will have access to a drug called Iplex, which has been unavailable to them since 2007.
Iplex is made by the Richmond-based company Insmed, and had been used in an off-label fashion by some patients with amyotrophic lateral sclerosis, commonly known as Lou Gehrig's disease.
It was taken off the U.S. market in 2007 when California-based Genentech sued Insmed, saying it used a component licensed exclusively to another drug company, Tercica.
That resulted in an outcry from patients who were using Iplex and those who wanted to do so. Attorneys at the local office of Richmond-based Williams Mullen got involved with the effort to free the drug for distribution after hearing that Josh Thompson, a Virginia Beach resident and 34-year-old son of developer Bruce Thompson, wanted access to the drug.
Josh Thompson has ALS, in which patients progressively lose control of their voluntary muscles, such as the ones that control swallowing and breathing, and eventually become paralyzed. Life expectancy after diagnosis is usually two to seven years.
In response to patients' concerns, Genentech, Tercica and Insmed signed a letter of intent in November freeing Insmed to supply the drug if it received regulatory permission from the U.S. Food and Drug Administration.
The FDA announced Tuesday on its Web site that people with ALS would have access to the drug under "Investigational New Drug" applications. Those who had requested "compassionate use" by last Friday will be able to receive the drug.
The remaining supply of Iplex, which is limited, will be used by Insmed to conduct a clinical trial.
Kathy Thompson, mother of Josh Thompson, said her son's Virginia Beach doctor called Tuesday to let them know the FDA had released use of the drug for her son, who had made an investigational application last year. He hopes to have the drug within two weeks.
"We are cautiously excited," Kathy Thompson said.
She said her son did a wheelchair dance in response to the news.
The statement on the FDA Web site said Insmed does not have enough of the drug to treat every patient who may request it, so the federal regulatory agency tried to find a fair way to provide access to the drug.
"FDA has attempted to balance the needs of individual patients who are desperately seeking treatment options for this devastating disease with the need to learn if the drug is in fact beneficial, or harmful, in treating patients with ALS," the statement reads.
People who have not already filed an application for use of the drug but want to participate in the clinical trial will be randomly assigned to receive the drug through a lottery system.
Currently the drug is used for a growth hormone deficiency but has not had rigorous testing among patients with ALS. Health providers who administer the drug for ALS patients are responsible for informing them of the risks.
For more information about enrolling in the clinical trial lottery, contact Insmed at (804) 565-3083 or iplex-als@insmed.com.
Elizabeth Simpson, (757) 446-2635, elizabeth.simpson@pilotonline.com
